THX Pharma and Exeltis Secure EMA Paediatric Committee Approval for TX01, Unlocking PUMA Pathway for Niemann-Pick Type C Disease
核心洞察
The EMA's Paediatric Committee (PDCO) issued a positive Opinion on the Paediatric Investigation Plan (PIP) for TX01 in Niemann-Pick type C disease (搜索), a critical regulatory milestone.
The approved PIP clears the way for a Paediatric Use Marketing Authorisation (PUMA) submission, which Exeltis (搜索) plans to file in Q1 2027.
A PUMA would provide up to 10 years of European market protection and a single marketing authorisation valid across all 27 EU Member States plus Iceland, Liechtenstein, and Norway.
THX Pharma (搜索) (Theranexus) and Exeltis (搜索), a global pharmaceutical company within the Insud Pharma (搜索) group, announced today that the European Medicines Agency's Paediatric Committee (PDCO) has issued a positive Opinion on the Paediatric Investigation Plan (PIP) for TX01 in Niemann-Pick type C disease (搜索). The EMA's subsequent Decision confirms the alignment of the TX01 development programme with European paediatric regulatory standards and unlocks a Paediatric Use Marketing Authorisation (PUMA) registration pathway in the European Union. Exeltis intends to file the PUMA application in Q1 2027.
Mathieu Charvériat, Chairman and Chief Executive Officer of THX Pharma (搜索), stated: "Securing the PDCO's positive Opinion on the PIP is one of the most discriminating regulatory milestones a paediatric drug development programme can achieve in Europe. It validates the scientific and clinical foundations of TX01 and, equally importantly, opens the PUMA pathway – a uniquely well-suited regulatory framework that combines a focused dossier, a centralised procedure and up to 10 years of paediatric market protection."
A Decisive Regulatory Gate Cleared
The PIP serves as the regulatory instrument governing paediatric development in the European Union. The PDCO's positive Opinion confirms that the agreed quality, non-clinical, and clinical development package — including the proposed paediatric formulation strategy for TX01 — is appropriate to support the targeted indications and age subsets, and that the timing and design of the studies are acceptable to the Agency.
An approved PIP is a regulatory prerequisite for any PUMA submission. With this Decision, THX Pharma (搜索) and Exeltis (搜索) now have a clear and de-risked path to file a centralised PUMA application with the EMA. Once granted, this would enable a single marketing authorisation valid across all 27 EU Member States plus Iceland, Liechtenstein, and Norway.
The PUMA Pathway: Tailored for Paediatric Innovation
The PUMA is a dedicated marketing authorisation route created by the EU Paediatric Regulation for medicines developed exclusively for paediatric use, based on active substances already authorised in adults. For TX01, the PUMA framework offers a uniquely favourable combination of regulatory and commercial advantages, including up to 10 years of paediatric market protection — comparable in scope to orphan exclusivity — and a centralised EU procedure delivering a single authorisation with one harmonised Summary of Product Characteristics and label across all participating countries.
The pathway also strengthens the product's position in pricing and reimbursement negotiations, as European health technology assessment bodies increasingly recognise dedicated paediatric formulations and PUMA status as a differentiating value driver in ultra-rare indications.
Addressing Unmet Need in Lysosomal Storage Disorders
TX01 is a novel paediatric formulation of an already approved compound, developed to address the specific needs of children living with Gaucher disease (搜索) and Niemann-Pick type C disease (搜索) — two rare, progressive, and severely debilitating lysosomal storage disorders for which paediatric therapeutic options remain limited.
This regulatory achievement builds directly on the successful completion of the first key milestone in the THX Pharma (搜索)–Exeltis (搜索) partnership, announced on October 14, 2025, and confirms that both companies remain on track to deliver TX01 to patients as planned. The companies confirm their objective of commercial launch in Europe in 2027, followed by launches in Latin America and selected Middle Eastern markets.
About the Companies
THX Pharma (搜索) (Theranexus) is a pharmaceutical company specializing in treatments for rare neurological diseases. Beyond TX01, its pipeline includes Batten-1, targeting the juvenile form of Batten disease (搜索), and an innovative antisense oligonucleotide platform codeveloped with leading research laboratories. THX Pharma is listed on Euronext Growth Paris (FR0013286259 – ALTHX).
Exeltis (搜索), part of the Insud Pharma (搜索) group, holds a leading position in women's health and is a key player in central nervous system therapies. Insud Pharma operates across the entire pharmaceutical value chain, with a presence in approximately 50 countries, 10,000 employees worldwide, and 20 production sites.